Cambridge, MA-based Genzyme, owned by French drug giant Sanofi (NYSE: SNY) said today its experimental drug to treat multiple sclerosis, alemtuzumab (Lemtrada), reduced relapse rates by 49 percent and dropped the risk of sustained worsening of disabilities by 42 percent in patients treated in a pivotal, Phase 3 trial. The drug was tested in a head-to-head trial against a form of interferon sold under the brand name Rebif by EMD Serono and Pfizer (NYSE: PFE). Genzyme plans to file for FDA approval of the drug in the first quarter of 2012, and it will be reviewed in an expedited manner by the FDA, which has granted it “fast track” status.
Genzyme scientists are particularly encouraged by how long-lasting the drug’s effects are proving to be. In the trial, one group of patients received alemtuzumab as a daily IV injection for five days, but then didn’t receive any medication for a year, at which point they were given the drug again for three days. The patients in the comparator group, by contrast, had to take interferon “subcutaneously” (injected under the skin) three times a week throughout the two-year trial period. “The contrast is obvious,” says Michael Panzara, therapeutic area head for multiple sclerosis, immune diseases, and neurology for Genzyme. “Even though our drug is active for a short period of time, the change it creates in the immune environment persists.” Panzara points out that in a previously reported five-year trial, patients only got two courses of alemtuzumab, but its efficacy lasted for the entire duration of the trial.
Alemtuzumab is an antibody that works by targeting a protein on cell surfaces called CD52. Genzyme’s research suggests that the drug depletes the T and B cells believed to be responsible for cell damage in MS. “When the cells come back, they are different,” Panzara says, “and we believe that leads to the prolonged effect.”
Patients in the trial suffered from a form of the disease called relapsing-remitting multiple sclerosis, and they had previously failed to respond to other therapies. Patients with this type of MS suffer episodes of weakness, sensory changes, severe fatigue, and other symptoms that “cause neurological deficits that do not go away,” Panzara says. “It knocks them out of work, it limits their ability to interact with their children.”
David Meeker, who became CEO of the Genzyme unit on November 1, believes the efficacy and dosing regimen offered by alemtuzumab in the trial are unprecedented. “This trial focuses on a population of patients who are underserved,” he says. “They continued to progress on other therapies. We’re confident we will be able to offer a highly differentiated offering for these patients.”
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